Duchenne Muscular Dystrophy & 21st Century Cures Legislation

Tell the Senate to Act!

As you know, PPMD has been helping to lead national advocacy efforts around the 21st Century Cures/ Senate Innovation legislation for more than two years and we now. You'll recall that this legislation has been passed in the House by am overwhelming majority vote and now has a very small window of opportunity in which the Senate can act to finalize this legislation so that both chambers can ‘conference’ these bills, come to agreement, and sign this game-changing legislation into law. THIS is where the rubber meets the road for this bill - and time is of the essence.

This package includes many provisions that are critical to our community including our Patient Focused Impact Assessment Act (PFIA, S 1597), provisions that create expedited pathways for follow-on exon development, data-sharing incentives, expanded access requirements, and much more. In addition, federal funding for the federal agencies that serve as the life blood to our research and research infrastructure pipelines would receive essential boosts including NIH and FDA.

We cannot afford for this bill to not be signed into law by this Congress.

We are turning to you, our powerful Duchenne community, to take action to bring this bill to the Senate floor. We must stay engaged throughout the remainder of the summer. We know that you are all super busy — so this can be quick and easy.

Please take a minute to send the following action Alert to your two Senators.


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The Duchenne Community Urges The U.S. Senate To Pass the Medical Innovations Package with Key Provisions

Dear [Decision Maker],

Our Duchenne community urges you to act on the Senate Medical Innovations package. The companion to 21st Century Cures in the House. Contained within this package are many provisions that matter to our community, including the Patient Focused Impact Assessment Act (S.1597) and the Advancing Targeted Therapies for Rare Diseases Act of 2015 (S.2030), both passed by the Senate HELP committee.

PFIA (S.1597) asks for an assessment from the Secretary as to whether FDA reviewers utilized Patient Focused Drug Development (PFDD) tools such as benefit/risk data, draft or final guidances, patient experience data, patient preference data, and other items throughout product reviews. In addition to the assessment, the legislation directs FDA to issue guidance to further clarify and inform development of PFDD tools created by patient-industry collaborations.

The Targeted Therapies provision (S.2030) clarifies FDA's existing authority to leverage data previously used in the approval of a targeted drug when approving a new therapy that "incorporates or utilizes the same or similar genetically targeted technology, or the same variant protein targeted technology". This authority is critically important to accelerating the development of treatments and cures for the numerous devastating rare diseases or subsets of rare diseases that otherwise have little hope of a treatment or cure due to their extremely small population size.

These bills represent real hope for our Duchenne community in moving the needle for rare disease drug development.

Please keep my voice and the voices of rare disease patients across this great country in mind while this critical package moves through the legislative process.

[Your Name]
[Your Address]
[City, State ZIP]
[Your Email]